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Engaging patients in research is encouraged. Yet, few qualitative research studies measure the impact of patient engagement (PE) at each research stage and from the point of view of all stakeholders. We studied two project groups to fill this research gap. Patient research partners (PRPs), researchers, and clinicians comprised each group. The two groups had the same objective to design and conduct a qualitative research project. We measured PE using three PE evaluation tools. All project group members were asked to complete the tools at three time points in the study. PRPs influenced and impacted many aspects of their project including the design, process, and approach of the qualitative research.

IMPORTANCE: For adults with appendicitis, several randomized clinical trials have demonstrated that antibiotics are an effective alternative to appendectomy. However, it remains unknown how the characteristics of patients in such trials compare with those of patients who select their treatment and whether outcomes differ. OBJECTIVE: To compare participants in the Comparison of Outcomes of Antibiotic Drugs and Appendectomy (CODA) randomized clinical trial (RCT) with a parallel cohort study of participants who declined randomization and self-selected treatment. DESIGN, SETTING, AND PARTICIPANTS: The CODA trial was conducted in 25 US medical centers. Participants were enrolled between May 3, 2016, and February 5, 2020; all participants were eligible for at least 1 year of follow-up, with all follow-up ending in 2021. The randomized cohort included 1094 adults with appendicitis; the self-selection cohort included patients who declined participation in the randomized group, of whom 253 selected appendectomy and 257 selected antibiotics. In this secondary analysis, characteristics and outcomes in both self-selection and randomized cohorts are described with an exploratory analysis of cohort status and receipt of appendectomy. INTERVENTIONS: Appendectomy vs antibiotics. MAIN OUTCOMES AND MEASURES: Characteristics among participants randomized to either appendectomy or antibiotics were compared with those of participants who selected their own treatment. RESULTS: Clinical characteristics were similar across the self-selection cohort (510 patients; mean age, 35.8 years [95% CI, 34.5-37.1]; 218 female [43%; 95% CI, 39%-47%]) and the randomized group (1094 patients; mean age, 38.2 years [95% CI, 37.4-39.0]; 386 female [35%; 95% CI, 33%-38%]). Compared with the randomized group, those in the self-selection cohort were less often Spanish speaking (n = 99 [19%; 95% CI, 16%-23%] vs n = 336 [31%; 95% CI, 28%-34%]), reported more formal education (some college or more, n = 355 [72%; 95% CI, 68%-76%] vs n = 674 [63%; 95% CI, 60%-65%]), and more often had commercial insurance (n = 259 [53%; 95% CI, 48%-57%] vs n = 486 [45%; 95% CI, 42%-48%]). Most outcomes were similar between the self-selection and randomized cohorts. The number of patients undergoing appendectomy by 30 days was 38 (15.3%; 95% CI, 10.7%-19.7%) among those selecting antibiotics and 155 (19.2%; 95% CI, 15.9%-22.5%) in those who were randomized to antibiotics (difference, 3.9%; 95% CI, −1.7% to 9.5%). Differences in the rate of appendectomy were primarily observed in the non-appendicolith subgroup. CONCLUSIONS AND RELEVANCE: This secondary analysis of the CODA RCT found substantially similar outcomes across the randomized and self-selection cohorts, suggesting that the randomized trial results are generalizable to the community at large. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT02800785

Lung cancer screening, despite its proven mortality benefit, remains vastly underutilized. Previous studies examined knowledge, attitudes, and beliefs to better understand the reasons underlying the low screening rates. These investigations may have limited generalizability because of traditional participant recruitment strategies and examining only subpopulations eligible for screening. The current study used crowdsourcing to recruit a broader population to assess these factors in a potentially more general population.

IMPORTANCE: Prognostic understanding can shape patients’ treatment goals and preferences. Patients undergoing dialysis in the United States have limited life expectancy and may receive end-of-life care directed at life extension. Little is known about their prognostic expectations. OBJECTIVE: To understand the prognostic expectations of patients undergoing dialysis and how these relate to care planning, goals, and preferences. DESIGN, SETTING, AND PARTICIPANTS: Cross-sectional survey study of 993 eligible patients treated with regular dialysis at 31 nonprofit dialysis facilities in 2 metropolitan areas (Seattle, Washington, and Nashville, Tennessee) between April 2015 and October 2018. After a pilot phase, 1431 eligible patients were invited to participate (response rate, 69.4%). To provide a context for interpreting survey participants’ prognostic estimates, United States Renal Data System standard analysis files were used to construct a comparison cohort of 307 602 patients undergoing in-center hemodialysis on January 1, 2006, and followed for death through July 31, 2017. Final analyses for this study were conducted between November 2018 and March 2019. EXPOSURES: Responses to the question “How long would you guess people your age with similar health conditions usually live?” (<5 years, 5-10 years, >10 years, or not sure). MAIN OUTCOMES AND MEASURES: Self-reported (1) documentation of a surrogate decision-maker, (2) documentation of treatment preferences, (3) values around life prolongation, (4) preferences for receipt of cardiopulmonary resuscitation and mechanical ventilation, and (5) desired place of death. RESULTS: Of the 993 survey respondents, the mean (SD) age was 62.6 (13.9) years, and 438 (44.1%) were women. Overall, 111 (11.2%) survey respondents selected a prognosis of fewer than 5 years, 150 (15.1%) respondents selected 5 to 10 years, 328 (33.0%) respondents selected more than 10 years, and 404 (40.7%) were not sure. By comparison, 185 427 (60.3%) prevalent US in-center patients undergoing hemodialysis died within 5 years, 58 437 (19.0%) died within 5 to 10 years, and 63 738 (20.7%) lived more than 10 years. In analyses adjusted for participant characteristics, survey respondents with a prognostic expectation of more than 10 years (vs <5 years) were less likely to report documentation of a surrogate decision-maker (adjusted odds ratio [aOR], 0.6; 95% CI, 0.4-0.9) and treatment preferences (aOR, 0.4; 95% CI, 0.2-0.6) and to value comfort over life extension (aOR, 0.1; 95% CI, 0.04-0.3), and were more likely to want cardiopulmonary resuscitation (aOR, 5.3; 95% CI, 3.2-8.7) and mechanical ventilation (aOR, 2.2; 95% CI, 1.2-3.7). The respondents who reported that they were not sure about prognosis had similar associations. CONCLUSIONS AND RELEVANCE: Uncertain and overly optimistic prognostic expectations may limit the benefit of advance care planning and contribute to high-intensity end-of-life care in patients undergoing dialysis.

IMPORTANCE: Use of antibiotics for the treatment of appendicitis is safe and has been found to be noninferior to appendectomy based on self-reported health status at 30 days. Identifying patient characteristics associated with a greater likelihood of appendectomy within 30 days in those who initiate antibiotics could support more individualized decision-making. OBJECTIVE: To assess patient factors associated with undergoing appendectomy within 30 days of initiating antibiotics for appendicitis. DESIGN, SETTING, AND PARTICIPANTS: In this cohort study using data from the Comparison of Outcomes of Antibiotic Drugs and Appendectomy (CODA) randomized clinical trial, characteristics among patients who initiated antibiotics were compared between those who did and did not undergo appendectomy within 30 days. The study was conducted at 25 US medical centers; participants were enrolled between May 3, 2016, and February 5, 2020. A total of 1552 participants with acute appendicitis were randomized to antibiotics (776 participants) or appendectomy (776 participants). Data were analyzed from September 2020 to July 2021. EXPOSURES: Appendectomy vs antibiotics. MAIN OUTCOMES AND MEASURES: Conditional logistic regression models were fit to estimate associations between specific patient factors and the odds of undergoing appendectomy within 30 days after initiating antibiotics. A sensitivity analysis was performed excluding participants who underwent appendectomy within 30 days for nonclinical reasons. RESULTS: Of 776 participants initiating antibiotics (mean [SD] age, 38.3 [13.4] years; 286 [37%] women and 490 [63%] men), 735 participants had 30-day outcomes, including 154 participants (21%) who underwent appendectomy within 30 days. After adjustment for other factors, female sex (odds ratio [OR], 1.53; 95% CI, 1.01-2.31), radiographic finding of wider appendiceal diameter (OR per 1-mm increase, 1.09; 95% CI, 1.00-1.18), and presence of appendicolith (OR, 1.99; 95% CI, 1.28-3.10) were associated with increased odds of undergoing appendectomy within 30 days. Characteristics that are often associated with increased risk of complications (eg, advanced age, comorbid conditions) and those clinicians often use to describe appendicitis severity (eg, fever: OR, 1.28; 95% CI, 0.82-1.98) were not associated with odds of 30-day appendectomy. The sensitivity analysis limited to appendectomies performed for clinical reasons provided similar results regarding appendicolith (adjusted OR, 2.41; 95% CI, 1.49-3.91). CONCLUSIONS AND RELEVANCE: This cohort study found that presence of an appendicolith was associated with a nearly 2-fold increased risk of undergoing appendectomy within 30 days of initiating antibiotics. Clinical characteristics often used to describe severity of appendicitis were not associated with odds of 30-day appendectomy. This information may help guide more individualized decision-making for people with appendicitis.

Stakeholder engagement in comparative effectiveness research continues to gain national attention. While various methods are used to gather stakeholder expertise and form recommendations, evaluation of the stakeholder experience is often missing. The lack of evaluation prohibits assessing how effective and meaningful engagement practices are for enhancing research efforts and limits the ability to identify areas for future improvement. We propose that an evaluation plan of engagement processes be developed before stakeholder involvement begins and be required as part of a request for proposal or research grant where stakeholder input is being sought. Furthermore, we recommend the inclusion of six meta-criteria that represent normative goals of multiple studies: respect, trust, legitimacy, fairness, competence and accountability. To aid in the development of future evaluations, we have developed definitions for and matched specific examples of measuring each meta-criterion to serve a guide for others in the field.

Aims:The Center for Comparative Effectiveness Research in Cancer Genomics completed a 2-year stakeholder-guided process for the prioritization of genomic tests for comparative effectiveness research studies. We sought to evaluate the effectiveness of engagement procedures in achieving project goals and to identify opportunities for future improvements. Materials & methods:The evaluation included an online questionnaire, one-on-one telephone interviews and facilitated discussion. Responses to the online questionnaire were tabulated for descriptive purposes, while transcripts from key informant interviews were analyzed using a directed content analysis approach. Results:A total of 11 out of 13 stakeholders completed both the online questionnaire and interview process, while nine participated in the facilitated discussion. Eighty-nine percent of questionnaire items received overall ratings of agree or strongly agree; 11% of responses were rated as neutral with the exception of a single rating of disagreement with an item regarding the clarity of how stakeholder input was incorporated into project decisions. Recommendations for future improvement included developing standard recruitment practices, role descriptions and processes for improved communication with clinical and comparative effectiveness research investigators. Conclusions:Evaluation of the stakeholder engagement process provided constructive feedback for future improvements and should be routinely conducted to ensure maximal effectiveness of stakeholder involvement.

Aims:Stakeholder engagement is fundamental to comparative effectiveness research (CER), but lacks consistent terminology. This paper aims to define stakeholder engagement and present a conceptual model for involving stakeholders in CER. Materials & methods:The definitions and model were developed from a literature search, expert input and experience with the Center for Comparative Effectiveness Research in Cancer Genomics, a proof-of-concept platform for stakeholder involvement in priority setting and CER study design. Results:Definitions for stakeholder and stakeholder engagement reflect the target constituencies and their role in CER. The ‘analytic-deliberative’ conceptual model for stakeholder engagement illustrates the inputs, methods and outputs relevant to CER. The model differentiates methods at each stage of the project; depicts the relationship between components; and identifies outcome measures for evaluation of the process. Conclusion:While the definitions and model require testing before being broadly adopted, they are an important foundational step and will be useful for investigators, funders and stakeholder groups interested in contributing to CER.

The ABCD system was developed to predict early stroke risk after transient ischemic attack. Incorporation of brain imaging findings has been suggested, but reports have used inconsistent methods and been underpowered. We therefore performed an international, multicenter collaborative study of the prognostic performance of the ABCD2score and brain infarction on imaging to determine the optimal weighting of infarction in the score (ABCD2I).

IMPORTANCE: Although people receiving maintenance dialysis have limited life expectancy and a high burden of comorbidity, relatively few studies have examined spirituality and religious beliefs among members of this population. OBJECTIVE: To examine whether there is an association between the importance of religious or spiritual beliefs and care preferences and palliative care needs in people who receive dialysis. DESIGN, SETTING, AND PARTICIPANTS: A cross-sectional survey study was conducted among adults who were undergoing maintenance dialysis at 31 facilities in Seattle, Washington, and Nashville, Tennessee, between April 22, 2015, and October 2, 2018. The survey included a series of questions assessing patients’ knowledge, preferences, values, and expectations related to end-of-life care. Data were analyzed from February 12, 2020, to April 21, 2021. EXPOSURES: The importance of religious or spiritual beliefs was ascertained by asking participants to respond to this statement: “My religious or spiritual beliefs are what really lie behind my whole approach to life.” Response options were definitely true, tends to be true, tends not to be true, or definitely not true. MAIN OUTCOMES AND MEASUREMENTS: Outcome measures were based on self-reported engagement in advance care planning, resuscitation preferences, values regarding life prolongation, preferred place of death, decision-making preference, thoughts or discussion about hospice or stopping dialysis, prognostic expectations, and palliative care needs. RESULTS: A total of 937 participants were included in the cohort, of whom the mean (SD) age was 62.8 (13.8) years and 524 (55.9%) were men. Overall, 435 (46.4%) participants rated the statement about religious or spiritual beliefs as definitely true, 230 (24.6%) rated it as tends to be true, 137 (14.6%) rated it as tends not to be true, and 135 (14.4%) rated it as definitely not true. Participants for whom these beliefs were more important were more likely to prefer cardiopulmonary resuscitation (estimated probability for definitely true: 69.8% [95% CI, 66.5%-73.2%]; tends to be true: 60.8% [95% CI, 53.4%-68.3%]; tends not to be true: 61.6% [95% CI, 53.6%-69.6%]; and definitely not true: 60.6% [95% CI, 52.5%-68.6%]; P for trend?=?.003) and mechanical ventilation (estimated probability for definitely true: 42.6% [95% CI, 38.1%-47.0%]; tends to be true: 33.5% [95% CI, 25.9%-41.2%]; tends not to be true: 35.1% [95% CI, 27.2%-42.9%]; and definitely not true: 27.9% [95% CI, 19.6%-36.1%]; P for trend?=?.002) and to prefer a shared role in decision-making (estimated probability for definitely true: 41.6% [95% CI, 37.7%-45.5%]; tends to be true: 35.4% [95% CI, 29.0%-41.8%]; tends not to be true: 36.0% [95% CI, 26.7%-45.2%]; and definitely not true: 23.8% [95% CI, 17.3%-30.3%]; P for trend?=?.001) and were less likely to have thought or spoken about stopping dialysis. These participants were no less likely to have engaged in advance care planning, to value relief of pain and discomfort, to prefer to die at home, to have ever thought or spoken about hospice, and to have unmet palliative care needs and had similar prognostic expectations. CONCLUSIONS AND RELEVANCE: The finding that religious or spiritual beliefs were important to most study participants suggests the value of an integrative approach that addresses these beliefs in caring for people who receive dialysis.

IMPORTANCE: Intermittent claudication (IC) is the most common presentation of infrainguinal peripheral artery disease. Both medical and revascularization interventions for IC aim to increase walking comfort and distance, but there is inconclusive evidence of the comparative benefit of revascularization given the possible risk of limb loss. OBJECTIVE: To compare the effectiveness of a medical (walking program, smoking cessation counseling, and medications) vs revascularization (endovascular or surgical) intervention for IC in the community, focusing on outcomes of greatest importance to patients. DESIGN, SETTING, AND PARTICIPANTS: Longitudinal (12-month follow-up) prospective observational cohort study conducted between July 3, 2011, and November 5, 2014, at 15 clinics associated with 11 hospitals in Washington State. Participants were 21 years or older with newly diagnosed or established IC. INTERVENTIONS: Medical or revascularization interventions. MAIN OUTCOMES AND MEASURES: Primary end points were 12-month change scores on the distance, speed, and stair-climb domains of the Walking Impairment Questionnaire (score range, 0-100). Secondary outcomes were change scores on the Walking Impairment Questionnaire pain domain (score range, 0-100), Vascular Quality of Life Questionnaire (VascuQol) (score range, 1-7), European Quality of Life–5 Dimension Questionnaire (EQ-5D) (score range, 0-1), and Claudication Symptom Instrument (CSI) (score range, 0-4). RESULTS: A total of 323 adults were enrolled, with 282 (87.3%) in the medical cohort. At baseline, the mean duration of disease was longer for participants in the medical cohort, while those in the revascularization cohort reported more severe disease. Other characteristics were well balanced. At 12 months, change scores in the medical cohort reached significance for the following 3 outcomes: speed (5.9; 95% CI, 0.5-11.3; P = .03), VascuQol (0.28; 95% CI, 0.08-0.49; P = .008), and EQ-5D (0.038; 95% CI, 0.011-0.066; P = .006). In the revascularization cohort, there were significant improvements in the following 7 outcomes: distance (19.5; 95% CI, 7.9-31.0; P = .001), speed (12.1; 95% CI, 1.4-22.8; P = .03), stair climb (11.4; 95% CI, 1.3-21.5; P = .03), pain (20.7; 95% CI, 11.0-30.4; P < .001), VascuQol (1.10; 95% CI, 0.80-1.41; P < .001), EQ-5D (0.113; 95% CI, 0.067-0.159; P < .001), and CSI (−0.63; 95% CI, −0.96 to −0.31; P < .001). Relative improvements (percentage changes) at 12 months in the revascularization cohort over the medical cohort were observed as follows: distance (39.1%), speed (15.6%), stair climb (9.7%), pain (116.9%), VascuQol (41%), EQ-5D (18%), and CSI (13.5%). CONCLUSIONS AND RELEVANCE: Among patients with IC, those in the revascularization cohort had significantly improved function (Walking Impairment Questionnaire), better health-related quality of life (VascuQol and EQ-5D), and fewer symptoms (CSI) at 12 months compared with those in the medical cohort, providing important information to inform treatment strategies in the community.

Platelet activation markers include activated glycoprotein (GP) IIbIIIa, P-selectin expression, platelet microparticles determined by flow cytometry, shear-induced platelet aggregation (SIPA), measured in a Sipagreg device that reproduces rheological conditions of stenotic or small arteries. We also studied endothelial activation markers including von Willebrand factor antigen (vWF), homocystein and high sensitivity C-reactive Protein (hsCRP). All these parameters were measured in 74 consecutive patients with recent lacunar stroke, in whom detectable large artery atherosclerosis or cardiac embolism have been ruled out, and in 74 population-based controls with no stroke history, matched for age, sex, hypertension, and diabetes. All patients were treated by anti-aggregant therapy, 15 (20%) were treated by clopidogrel (75 mg/d) and the remaining 59 cases received only acetylsalicylic acid (75 to 160 mg/d). Repeated blood samples were collected at one- and three-months after symptom onset.One month after symptom onset, patients had similar levels of platelet activation compared to matched controls (p>0.40 for all-comparisons), including platelet membrane P-selectin with a median value of 1.3% and an interquartile range (IQR) (0.7–2.8) in patients and 1.1% (0.6–2.2) in controls, p=0.37), platelet activated GPIIbIIIa reached 2.9% (0.9–6.7) in patients and 2.8% (0.7–10.2) in controls, p=0.94. Platelet-derived microparticles were 5067/μL of blood (3720–7490) and 5035 (3530–8150) p=0.63l, in patients and controls, respectively. SIPA values at 4000 sec-1 were in the normal range in patients and controls, with no difference between the two groups: the median value of platelet aggregation was 22% (12–37%) in cases and 23% (7–45%) in controls (p=0.96), respectively. Endothelial activation parameters were increased in patients in comparison to controls (vWF p=0.002 and homocystein/creatinemia p=0.025) The median value of vWF antigen was 142% (114–182%) in cases and 122% (97–150%) in controls (p=0.002). The median value of ratio homocysteinemia/creatinemia was 0.151 (0.119–0.187) in cases and 0.134 (0.111–0.165) in controls (p=0.025). The median value of hsCRP was 2.7 mmol/l (1.5–6.0) in cases and 2.0 mmol/l (1.0–3.4) in controls (p=0.059). Level of hsCRP was slightly increased in patients compared to controls (p=0.059). At 3 months, significant decrease in vWF and hsCRP levels (median change in vWF=10%, p=0.004; median change in hsCRP=0.4 mg/L, p=0.02) was detected in patients. Homocystein level and all platelet parameters remained unchanged at this time compared to 1 month.Our results confirm that endothelial dysfunction is a major feature of lacunar stroke. One explanation could be an endothelial response to an acute systemic aggression by an unidentified agent. In contrast, chronic platelet activation, when compared to controls matched for age, sex and vascular risk factors, did not seem to play a central role in the pathophysiology of lacunar stroke.No relevant conflicts of interest to declare.

Several letters to the editor are presented in response to articles in previous issues including "Regaining Attitude" in the January 17, 2008 issue, "Mayencourt Still Considering Conservative Run" in the December 2007 issue, and "Christmas as a Gay Celebration" which features holiday celebration in the December 20, 2007 issue.